Trial: A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

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Quick Info

Status

Currently Recruiting

Phase

Trial Type

Interventional

Treatment Type

Drug Trials

Randomization

Not Available

Enrollment

Start Date

10/1/2021

Sponsor

Transposon Therapeutics, Inc.

Contact Information

Contact information unknown.

Locations

Belgium, Other

VIB-KU Leuven Center for Brain & Disease Research, Leuven, 3000, Belgium
Contact: Philip Van Damme, MD, PhD

France, Other

CHU Lille - CMRR Hôpital Roger Salengro, Lille, 59037, France
Contact: Florence Pasquier, MD, PhD

CHU Dupuytren, Limoges, Limoges, 87042, France
Contact: Philippe Couratier, MD, PhD

Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux, Paris, 75013, France
Contact: Richard Levy, MD, PhD

Germany, Other

Universitaetsklinikum Ulm - Klinik fuer Neurologie, Ulm, 89081, Germany
Contact: Albert C Ludolph, MD

Spain, Other

Hospital de la Santa Creu i Sant Pau, Barcelona, 08025, Spain
Contact: Alberto Lleo Bisa, MD, PhD

Hospital Universitario Vall d'Hebron, Barcelona, 08035ES, Spain
Contact: Raul Juntas Morales, MD

Complejo Hospitalario Universitario de Santiago (CHUS), Santiago de Compostela, 15706ES, Spain
Contact: Julio Pardo Fernandez, MD, PhD

Hospital Universitari I Politècnic La Fe, Valencia, 46026, Spain
Contact: Miquel Baquero Toledo, MD

United States, California

University of California San Diego, La Jolla, CA, 92037, United States
Contact: Dominic Ferrey, MD

University of California Irvine - ALS & Neuromuscular Center, Orange, CA, 92868, United States
Contact: Namita A Goyal, MD

University of California at San Francisco/UCSF, San Francisco, CA, 55455, United States
Contact: Adam Boxer, MD, PhD

United States, Maryland

John Hopkins University, Baltimore, MD, 21287, United States
Contact: Chiadi Onyike, M.B.B.S., MD

Johns Hopkins Outpatient Center, Baltimore, MD, 21287, United States
Contact: Jeffrey D Rothstein, MD, PhD

United States, Massachusetts

Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic, Boston, MA, 02114, United States
Contact: Doreen Ho, MD

United States, Minnesota

Mayo Family Clinic Northwest, Rochester, MN, 55905, United States
Contact: Bradley Boeve, MD

United States, New York

Hospital for Special Surgery, New York, NY, 10021, United States
Contact: Dale J Lange, MD

Columbia University Medical Center - The Neurological Institute of New York, New York, NY, 10032, United States
Contact: Lawrence Honig, MD, PhD

United States, North Carolina

The University of North Carolina at Chapel Hill, Department of Neurology, Chapel Hill, NC, 27599, United States
Contact: Andrea Bozoki, MD

Enrollment Criteria

Breathing Ability

Percent lung function (FVC) or (SVC)

>60% (SVC)

Months Since Onset

Number of months since first symptoms of ALS.

<3 Years

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?

Yes

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?

Yes

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?

Yes

Open Label

Yes

Update Notes

Site removed

11/8/2022

Sites added, protocol updates

4/22/2022

Sites added

3/11/2022

Sites added

2/8/2022

Study recruiting

12/6/2021

Outcome measures updates

8/17/2021

Other Information

Purpose

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

Eligibility

Inclusion Criteria:
- Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene - Has a reliable caregiver/informant to accompany the patient to all study visits For patients with ALS (with or without FTD):
- Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria - Onset of weakness within 3 years prior to Screening - Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position) - Able to perform reproducible pulmonary function tests.
- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 and score of 3 or 4 on Item #3 (swallowing) at Screening For patients with FTD:
- A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome - CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening Exclusion Criteria:
- Presence of other significant neurological or psychiatric disorders - History of clinically significant brain abnormality - Clinically significant medical illness - Tracheostomy or diaphragmatic pacing - Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable) - History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization

Details

This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with a long-term, open-label treatment phase in patients with C9ORF72 ALS and/or FTD. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open-label Treatment Period, and a Follow-up Visit 4 weeks post-treatment.

Collaborator(s)

Transposon Therapeutics, Inc.

Trial Protocol as Published on Clinicaltrials.gov

NCT04993755 (First Published: 7/19/2021)